gene therapy for cardiovascular disorders

... Trang 1CHAPTER 6Gene Therapy for Hematological Disorders CYNTHIA E DUNBAR, M.D and TONG WU, M.D. INTRODUCTION Hematopoietic cells are an attractive target for gene therapy for two main reasons ... forward into real clinical utility REQUIREMENTS FOR GENE TRANSFER INTO HEMATOPOIETIC CELLS Ex Vivo Versus in Vivo Gene Transfer Specific aspects of gene transfer techniques are advantageous for ... Targets and Applications for Gene Therapy of Hematopoietic or Immune System Disorders Cancer (TIL) AIDS (intracellular immunization) Antisense to oncogenes Tumor vaccines Suicide genes Growth factors...

Ngày tải lên: 10/04/2014, 22:10

... worldwide For these reasons, the liver has become an important target organ for gene therapy At the same time, certain circumstances make the liver an especially challengingtarget for gene therapy ... attempted so far GENERAL PRINCIPLES FOR HEPATIC GENE THERAPY There are two basic approaches for gene transfer into hepatocytes: ex vivo and invivo strategies (Fig 7.1) Ex vivo therapy requires ... the therapeutic gene Alternatively, dif-ferent viral vectors as well as liposomes can be used for ex vivo gene transfer Trang 3For in vivo gene therapy, the therapeutic or normal gene is introduced...

Ngày tải lên: 10/04/2014, 22:11

... 0-471-22387-5 (Electronic) Trang 2GENETIC MANIPULATION OF CARDIOVASCULAR TISSUEModulating Gene Expression in Cardiovascular Tissue Gene therapy can be defined as any manipulation of gene expression that influences ... the gene, preventing the translation into protein Another form of gene blockade is the use FIGURE 8.1 Gene therapy strategies See color insert (A) Gene transfer involves deliv-ery of an entire gene, ... angiogenic gene therapy may provide an alternative not currently available to a significant number of patients suffering from untreatable GENE THERAPY FOR ANGIOGENESIS 191 FIGURE 8.3 Combined gene...

Ngày tải lên: 10/04/2014, 22:11

... considered as anacquired genetic disorder As previously discussed, gene therapy holds considerablepotential for the treatment of hereditary and acquired genetic disorders Humangene therapy can be defined ... ultimate goal of gene therapy is to inhibit HIV-1 viral replication and theresulting AIDS pathogenesis For gene therapy of HIV infection to be successful, itwill be necessary to introduce genes that ... vivo is not plausible for gene therapy due to the vastnumber of cells that need to receive the therapeutic RNA An alternative approach to the delivery of antisense RNA for gene therapy isthe use...

Ngày tải lên: 10/04/2014, 22:11

... Trang 1Gene therapy for primary immune deficiencies: a Canadian perspective Xiaobai Xu1, Chetankumar S Tailor2 and Eyal Grunebaum1,3,4* Abstract The use of gene therapy (GT) for the treatment ... continue to carry significant complications Gene therapy Gene therapy (GT), i.e the use of genetic material to modify cells, has been investigated for numerous conditions since the development ... used for the correction of monogenetic disorders in post-mitotic tissues, while retroviral vectors can integrate into the host cell genome Therefore retroviruses are preferred for the stable gene...

Ngày tải lên: 04/12/2022, 10:38

... Trang 1R E V I E W A R T I C L EReceptor crosstalk Implications for cardiovascular function, disease and therapy Nduna Dzimiri Cardiovascular Pharmacology Laboratory, Biological and Medical Research ... any specific physiological role in cardiovascular signalling has been clearly defined for the majority of the crosstalk among these pathways,its existence in the cardiovascular system strongly points ... activation of Ras/Raf pathway to regulate gene expression essential for proliferation [43–47] (Fig 1) This process, traditionally conceived as an escape route for G-protein coupled receptors from...

Ngày tải lên: 21/02/2014, 15:20

... impor-tant for the resistance to possible introduction of genes using viral vectors or for the resistance to oncolytic gene therapy We believe that this finding can be of cru-cial interest for the ... viral gene therapy approaches Adenoviruses and Adeno-Associated viruses are used to deliver genes to tumor cells with the goal of modifying the phenotype, as for example, by introducing suicide genes ... type 5 forward primer 5 ’-AACCGAAGGCTGCATT-CACT, reverse primer 5 ’-ACCGCACAGGGTCTTAA-TAGAG Following denaturation at 96°C for 10 min, cycling conditions were 96°C for 15s, 60°C for 1 min for 40...

Ngày tải lên: 18/06/2014, 16:20

... and does not sensitive to radiotherapy and chemotherapy [2,3] There-fore, a novel therapy strategy against CCRCC needs to be developed In the past decade, gene therapy was stu-died world-widely ... [4] Thus, the use of gene therapy may be a new way to treat CCRCC In the field of cancer gene therapy, it is well known that the success of therapy is greatly dependent on the gene delivery vectors ... follows: first cycle at 95°C for 2 min, and then 30 cycles at 94°C for 45 s, 54°C for 1 min, 72°C for 1 min and a final extension cycle of 72°C for 5 min The house-keeping gene b-actin was taken as...

Ngày tải lên: 18/06/2014, 19:20

... and thus the former is the vector of choice for future gene therapy trials The number of gene therapy trials in RA is in sharp contrast to the more than 600 clinical trials of gene therapy in patients ... the application of gene therapy in RA, and the feasibility of gene therapy was recently demonstrated in phase I clinical trials Keywords: arthritis, cytokines, gene therapy, genetic synovectomy, ... safety of local gene therapy Gene therapy in RA is still in its adolescence, and more clinical studies must be initiated if it is to mature It is, however, imperative that gene therapy studies...

Ngày tải lên: 09/08/2014, 01:24

... progenitor cells are an essential ingredient for HIV gene therapy In view of the need for CD34 cells for HIV gene therapy as well as for other hematopoietic disorders, if one can pro-duce these cells ... lineages thus paving the way for utilization of these cells for hematopoietic cell therapy [20,27-29] For the effective utilization of hES-CD34 cells for HIV gene therapy, a number of parameters ... Stem Cell-based Gene Therapy for HIV/AIDS Anticancer Res 2003, 23:1997-2005. 14. Michienzi A, Castanotto D, Lee N, Li S, Zaia JA, Rossi JJ: RNA-medi-ated inhibition of HIV in a gene therapy setting...

Ngày tải lên: 13/08/2014, 09:21

... virus These results reveal potential pitfalls for future developments of gene therapy strategies of HIV infection Background Highly active antiretroviral therapy (HAART) effectively inhibits human ... infection could be increased by develop-ing a gene therapy strategy based on the modified produc-tion of IFN-β in genetically engineered lymphocytes [18] For this purpose, a retroviral vector derived ... observed, and these macaques maintained high CD4+ T-lymphocyte counts for at least 478 days [24] However, a gene therapy strategy for HIV infection would only be possible during the chronic phase...

Ngày tải lên: 13/08/2014, 13:20

... AccessProviral HIV-genome-wide and pol-gene specific Zinc Finger Nucleases: Usability for targeted HIV gene therapy Misaki Wayengera Correspondence: wmisaki@yahoo. com Unit of Genetics, Genomics & Theoretical ... http://www.ncbi.nlm.nih.gov/nuccore/9629357?report=fasta General discussion I report here SIV/HIV-pol gene and HIV-1 whole genome specific zinc finger nucleases, which are proposed for use towards targeted HIV gene therapy Specifically, ... of the HIV-1 genome For details, see additional file 3. Trang 7deliver genes ex vivo into bona fide stem cells, particularly hematopoietic stem cells,allowing for stable transgene expression upon...

Ngày tải lên: 13/08/2014, 16:20

... Corresponding author gliomabioluminescence imaginggene therapyherpes simplex virus type 1 thymidine kinaseluciferase Abstract Background: Suicide gene therapy employing the prodrug activating system ... insufficient transgene delivery to tumor cells Therefore, this study aimed at developing a strategy for real-time noninvasive in vivo monitoring of the activity of a therapeutic gene in brain tumor ... used for repetitive and noninvasive quantification of HSV-TK/ GCV mediated cell kill in vivo. Conclusion: This approach may represent a valuable tool for the in vivo evaluation of gene therapy...

Ngày tải lên: 14/08/2014, 19:22

... vectors for gene therapy-what are the risks? A review of retroviral pathogenesis and its relevance to retroviral vector-mediated gene delivery Donald S Anson*1,2,3 Address: 1 Department of Genetic ... clinical trial of γc mediated gene therapy for X-linked severe combined immunodeficiency (X-SCID) has proven the potential of retroviral mediated gene transfer for the treatment of inherited ... in activation of an onco-gene, in combination with the requirement for other genetic changes before a cancer eventuates Although pro-viral integration can also result in gene inactivation, inac-tivation...

Ngày tải lên: 14/08/2014, 19:22

... 1 of 11 (page number not for citation purposes) Genetic Vaccines and Therapy Open Access Research Human cytomegalovirus plasmid-based amplicon vector system for gene therapy Kutubuddin Mahmood, ... demonstrates that a foreign gene can be expressed in the context of a HCMV amplicon viral stock in infected HF cells. To test the utility of the HCMV amplicon in gene therapy or gene delivery, we ... hybridizes to a sequences. Genetic Vaccines and Therapy 2005, 3:1 http://www.gvt-journal.com/content/3/1/1 Page 8 of 11 (page number not for citation purposes) and expressing foreign genes in infected...

Ngày tải lên: 14/08/2014, 19:22

... Combining RNAi with this gene delivery system may enable us to develop RNAi for silencing EGFR into an effective therapy for NSCLC Published: 30 June 2005 Genetic Vaccines and Therapy 2005, 3:5 doi:10.1186/1479-0556-3-5 ... current gene transfer vector systems, inadequate weak promoters to drive transgene expression Therefore, so far, only three cancer gene therapy protocols had reached phase III trials before being ... cycle at 94°C for 2 min, and 45 cycles involving denaturation at 94°C for 10 s annealing at 53°C for 30 s and extension at 72°C for 40 s, followed by a final extension at 72°C for 10 min The...

Ngày tải lên: 14/08/2014, 19:22

... to serve as "Trojan horses" for the delivery of genes for cancer therapy Indeed, clostridial spores that were genetically manipulated to harbour genes for cancerstatic factors, prodrug ... consist of sev-eral aspects: one is from its transgene that encodes prod-rug converting enzymes for suicide-gene therapy or cytokines for immuno-gene therapy These are essentially the same as the ... vectors for cancer gene therapy At present, there are various gene therapy vector systems under development against cancer However, due to the complexity of the solid tumours involving angiogenesis,...

Ngày tải lên: 14/08/2014, 19:22

... as source for MSCs and NSCs in suicide gene therapy 113 5.1.1 Baculoviruses as gene therapy vectors for human cancer 113 5.1.2 HESC- derived MSCs and NSCs as gene delivery vehicles for 5.1.3 ... enabled the development of gene therapy, which introduces genetic material into cells for therapeutic purposes Originally, gene therapy was proposed to correct genetic disorders by transferring ... Trang 31compared to single gene expression or even the injection of two genes (Samaranayake 1.1.2 Gene carriers for cancer therapy Successful gene therapy relies largely on gene delivery efficiency...

Ngày tải lên: 09/09/2015, 10:08

... vectors in gene therapy for glioma Finally, this study demonstrated, for the first time, that human embryonic stem cells can provide a potentially unlimited source for glioma gene therapy Using ... for glioma gene therapy 137 4.4 Discussion 141 Chapter 5 Conclusions 146 Trang 10SUMMARY Malignant glioma remains one of the most lethal forms of cancer in humans However, current therapy for ... CELLULAR VEHICLES FOR GLIOMA GENE THERAPY ZHAO YING NATIONAL UNIVERSITY OF SINGAPORE 2008 Trang 2DEVELOPMENT OF NEW HUMAN STEM CELL-DERIVED CELLULAR VEHICLES FOR GLIOMA GENE THERAPY ZHAO YING...

Ngày tải lên: 11/09/2015, 09:00

... 2005), among which are suicide gene therapy, genetic immunotherapy, tumor suppressor gene or oncogene approaches, and anti-angiogenesis gene therapy Trang 14Suicide gene therapy is one of the commonly ... vector for gene therapy It is impossible to obtain success in gene therapy without effective gene delivery systems that can achieve high levels of therapeutic gene expression in targeted cells Gene ... accounting for 73% of the approved glioma gene therapy clinical trials (Barzon et al., 2006) As an attractive candidate for suicide gene therapy, the diphtheria toxin A-chain (DTA) gene has been...

Ngày tải lên: 01/10/2015, 11:39